Ptc Therapeutics Inc.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients; and Emflaza (deflazacort) for treating Duchenne muscular dystrophy. It is developing Translarna, which is in Phase II clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 for the treatment of patients with spinal muscular atrophy, as well as PTC596, an orally bioavailable and potent small molecule that has completed Phase I study, which targets solid tumor cell populations by reducing the activity and amount of BMI1. The company has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and research collaboration with CHDI Foundation, Inc. to discover and develop small-molecule therapeutics for Huntington's disease. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.